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FDA fast tracks ALS stem cell therapy

13 October 2014
Appeared in BioNews 775

The US Food and Drug Administration (FDA) has awarded 'fast-track' status to a potential stem cell therapy for amyotrophic lateral sclerosis (ALS), or motor neurone disease. The treatment, which uses modified stem cells from patients' bone marrow, is being developed by Israeli biotechnology company BrainStorm Cell Therapeutics, and is currently in a mid-stage clinical trial.

The FDA's fast-track programme aims to improve patient access to new therapies where current treatment options are insufficient.

BrainStorm chief executive Dr Tony Fiorino told Reuters: 'The receipt of fast-track designation from the FDA is an acknowledgement of the unmet medical need in ALS.' The Motor Neurone Disease Association in the UK estimates that seven out of every 100,000 people in the general population are living with the disease.

ALS - also known as Lou Gehrig's disease – is a degenerative condition that affects nerves in the spinal cord and brain. As the neurones die, the patient finds it more difficult to initiate and control muscle movement. Patients in the later stages of the disease, such as British physicist Professor Stephen Hawking, may become totally paralysed.

In the technique developed by BrainStorm, called NurOwn, stem cells are extracted from patients' bone marrow and then treated so that they are able to secrete 'neurotrophic factors' – proteins that are essential to the growth and survival of nerve cells. These 'MSC-NTF' cells are then injected back into the patient at the sites of the most severe nerve damage.

BrainStorm completed its second phase 2 clinical trial of NurOwn earlier this month, and the results will be published at the end of the year.

Professor Karussis Dimitrios, of the Hadassah Medical Centre in Jerusalem, who led the trial, said: 'I have now treated over 30 ALS patients with NurOwn in two clinical trials, as well as in several compassionate use treatments, and I am enthusiastic about its emerging safety and efficacy profile.'

According to Reuters, NurOwn is also being studied in a phase 2 clinical trial at three US hospitals.

Few cellular therapies have received FDA approval to date. Dr Fiorino told Reuters: 'What is so valuable about fast-track designation to a small company like BrainStorm is the opportunity to have increased meetings with and more frequent written communication from the FDA.'

BrainStorm gets FDA fast-track status for ALS stem cell therapy
Reuters |  7 October 2014
Press Release - October 6, 2014
BrainStorm Cell Therapeutics (press release) |  6 October 2014
US FDA grants Fast Track status to BrainStorm Cell Therapeutics for ALS treatment NurOwn
The Pharma Letter |  7 October 2014
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